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Repligen Receives Orphan Drug Designation from the FDA for RG2833 for Friedreich's Ataxia
Date:5/24/2010

macodynamic response of various biomarkers in peripheral blood to RG2833.  RG2833 is a new chemical entity, which is the subject of a composition of matter patent application, which if allowed, will remain in force until 2029 prior to any patent term extensions.  RG2833 has been developed in collaboration with scientists from The Scripps Research Institute and a broad network of international scientific thought leaders.  Repligen's research efforts have been partially funded with grants from the Muscular Dystrophy Association, the Friedreich's Ataxia Research Alliance, GoFAR and the National Ataxia Foundation.  We are also evaluating HDAC-3 inhibitors in animal models of Huntington's disease and cognition.

About Friedreich's Ataxia

Friedreich's ataxia is an inherited neurodegenerative disease caused by a single gene defect that results in inadequate production of the protein frataxin.  Low levels of frataxin lead to degeneration of both the nerves controlling muscle movements in the arms and legs and the nerve tissue in the spinal cord.  Symptoms of Friedreich's ataxia typically emerge between the ages of five and fifteen and often progress to severe disability, incapacitation or loss of life in early adulthood.  Preclinical studies have shown that specific HDAC inhibitors increase production of the protein frataxin, and may have the potential to arrest disease progression in patients with Friedreich's ataxia.  At present, there are no effective treatments for Friedreich's ataxia.

About Repligen Corporation

Repligen Corporation is a biopharmaceutical company focused on the development of novel therapeutics for neurological disorders.  In addition, we are the world's leading supplier of recombinant Protein A, the sales of w
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